According to a story from biotech-finances.com, the biopharmaceutical company miRagen Therapeutics, Inc., recently announced the release of data from a Phase 1 clinical trial of miR-155 inhibitor cobomarsen as a…
According to a story from ctvnews.ca, Paul Fitzpatrick of Ontario has been enduring severe eye pain following laser eye surgery for over twenty years before he committed suicide. The man's…
It was in 1993, just 25 years ago, that congress passed an act to require more diversity in clinical trials. Specifically, it required more female and ethnic minority representation. Since…
According to a story from PR Newswire, the 60th Annual Meeting of the American Society of Hematology has been generating a lot of press lately as many groundbreaking studies, research,…
According to a story from BioPortfolio, the global biopharmaceutical company Celgene Corporation recently announced the release of early data from the dose-escalation portion of a Phase 1/2 clinical trial of lisocabtagene…
Chronic myelomonocytic leukemia, or CMML, is a rare condition. However, it has similarities to myelodysplastic syndromes (MDS) and myeloproliferative neoplasms (MPN). That being said, it still a unique diagnosis, and…
Huntington's Disease is a neurodegenerative condition caused by a genetic mutation. It results in progressive decline of motor skills. It also affects cognitive function. Patients experience decline for 15-20 years…
Sickle cell disease (SCD) is a blood disorder. SCD patients have abnormal hemoglobin, or sickle hemoglobin, caused by a genetic mutation in the beta-chain. It results in deformed and inflexible…
Happy Wednesday! As the new month begins, we want to spotlight four articles. First, we have an incredible story about a Cushing's syndrome survivor and advocate and her talented service…
In the UK, a think tank determines access to rare disease drugs based on their perception of an individual's quality of life. That idea has also been developed in the…
Mantle cell lymphoma is caused by a genetic mutation which produces cancerous white blood cells. Of course, these are the cells which fight infection in a normal body. Therefore in…
According to a story from pm360online.com, the pharmaceutical company Zogenix Inc. recently announced that its experimental drug candidate ZX008, also known as Fintepla®, has performed well in the company's latest…
According to a story from The San Diego Union-Tribune, the Pancreatic Cancer Prevention and Screening Clinic at UC San Diego Moores Cancer center has opened for the first time this…
When Deborah, a New Yorker, flew over to Paris to do an internship for an architecture office, she never knew she would be flying across the world and meeting…
According to a story from BioPortfolio, the gene therapy company Rocket Pharmaceuticals has recently announced that the company's investigational gene therapy RP-L102 has earned both Fast Track and Regenerative Medicine…
According to a story from Reuters, the drug developer BioMarin is looking to make a name for itself in the hemophilia community in a very unique way. The company has…
According to a story from Grimsby Live, the family of six year old Melissa Stark, who has Williams syndrome, has transformed their house into a Santa Claus themed wonderland of…
Caregivers come from all walks of life but they share one common goal- taking care of someone they love who cannot fully take care of themselves. This may mean helping…
Lambert-Eaton Myasthenic Syndrome (LEMS) is an autoimmune disorder. It's a rare condition characterized by muscle weakness in the limbs. When the muscle weakness includes the respiratory muscles, the disease can…
Tiffany Rowe was 15 when she accomplished her biggest childhood dream. She met Michael Jackson at a live concert and actually got to dance on stage with him. Over…
According to a story from Medical Xpress, a team of scientists affiliated with the University of Granada have developed a new potential treatment for a form of mitochondrial disease that…
"It's funny to say grief as it is not a bereavement but you kind of are grieving the loss of the child you thought you had and the future they…
According to a story from Xconomy, the drug developer Vertex Pharmaceuticals recently announced that a three drug combination treatment for the rare lung disorder cystic fibrosis has performed well in…
On November 26, 2018, the FDA approved a new autoinjector for individuals with severe rheumatoid arthritis (RA), giant cell arteritis, and polyarticular or systemic juvenile idiopathic arthritis (aged 2 and older). It's…
At Patient Worthy, we are dedicated to stories about the latest in rare disease news and research, as well as posting stories from rare disease patients themselves. However, there are thousands…
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